Hereditary pulmonary alveolar proteinosis (herPAP) is a rare disease characterized by the slow build-up of lipo-protein material in the lungs due to the failure of highly specialized cells called macrophages, which usually eat away this material from the pulmonary air-space. Researchers demonstrate that a single transplantation of murine macrophages into the lungs of mice suffering […]

In a study of over 50 transcription factors, Tbx6 alone was able to stimulate mesoderm formation in laboratory-grown stem cells, and could cause those stem cells to become cardiovascular or musculoskeletal cells. Researchers found that this essential role of Tbx6 in mesoderm and cardiovascular specification is conserved from lower organisms to mammals. This research report […]

Researchers have created a drug delivery system (DDS) using a poly (ethylene glycol)-poly(lysine) block copolymer-ubenimex conjugate (PEG-b-PLys(Ube)). The use of this DDS has enabled an increase in the concentration of ubenimex in target CSCs. In addition, combined use of standard anticancer drugs significantly decreased CSCs.

Scientists have uncovered a reason for the uncanny likeness between cells in the most malignant cancers and the embryonic cells of the organ in which the cancer originated: cells in human basal-like breast cancers share features with the embryonic mammary (breast) stem cells that are the progenitors of all cell types in the mammary gland […]

A new comprehensive natural history study about Amish nemaline myopathy (ANM) in the Old Order Amish population focuses on the promise of gene therapy for this lethal disorder. Amish nemaline myopathy (ANM) is an infantile-onset muscle disease linked to a mutation of the TNNT1 gene. The study summarizes genealogical records, clinical data, and molecular reports […]

Researchers delayed signs of amyotrophic lateral sclerosis (ALS) in rodents by injecting them with a second-generation drug designed to silence the gene, superoxide dismutase 1 (SOD1). The results suggest the newer version of the drug may be effective at treating an inherited form of the disease caused by mutations in SOD1.